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Pharmacokinetics, Effectiveness and Tolerability of Prolonged-release Tacrolimus After Paediatric Kidney Transplantation

Phase 3
Recruiting
Conditions
Pediatric Kidney Disease
Interventions
Registration Number
NCT06057545
Lead Sponsor
University Hospital, Essen
Brief Summary

Recently, a new prolonged-release tablet version of tacrolimus (Envarsus®) using the so-called MeltDose™ (US Patent No. 7,217,431) drug-delivery technology has been approved as immunosuppressive medication for patients after kidney and liver transplantation in adults but not yet in children. Studies in adults proved that Envarsus® provides the same therapeutic effectiveness as the conventional immediate-release tacrolimus formulation (Prograf®) with improved bioavailability, a more consistent pharmacokinetic profile and reduced peak to trough which might result in reduced tacrolimus dosing and subsequently reduced CNI related toxicity. Furthermore, the once daily formulation might result in improved drug adherence.

The aim of this study is to assess pharmacokinetic profiles of Envarsus® as well as effectiveness and tolerability of this drug in children and adolescents ≥ 8 and ≤ 18 years of age.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
30
Inclusion Criteria
  1. caucasian paediatric kidney transplant recipients (single-organ recipients)
  2. aged ≥ 8 years but ≤ 18 years who are under tacrolimus (Prograf®) therapy and who are able to swallow tablets with a minimum dose of 0.75 mg / day Envarsus®
  3. not less than 6 months after transplantation
  4. stable kidney function (delta eGFR < 10 ml/min/1.73 m2 (CKID formula) over the last 3 months)
  5. women of childbearing potential and women without childbearing potential
  6. patient/parents/legal guardian(s) must be capable of understanding purpose and risks of the study
  7. signed informed consent obtained by patient and parents/legal guardians
Exclusion Criteria
  1. coefficient of variation of tacrolimus trough levels > 0.35 over the previous 6 months
  2. pregnancy/breast feeding
  3. instable kidney function
  4. hypersensitivity to any of the components of the medications used
  5. not eligible for any reason according to the investigator's valuation
  6. known positive HIV-1 or HCV test
  7. participation in another clinical trial (other investigational drugs or devices at the time of enrolment or within 30 days prior to enrolment)

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
Group A - Envarsus followed by PrografEnvarsus®4 weeks treatment sequence 1 (Envarsus) followed by 4 weeks treatment sequence 2 (Prograf)
Group B - Prograf followed by EnvarsusEnvarsus®4 weeks treatment sequence 2 (Prograf) followed by 4 weeks treatment sequence 1 (Envarsus)
Group B - Prograf followed by EnvarsusPrograf4 weeks treatment sequence 2 (Prograf) followed by 4 weeks treatment sequence 1 (Envarsus)
Group A - Envarsus followed by PrografPrograf4 weeks treatment sequence 1 (Envarsus) followed by 4 weeks treatment sequence 2 (Prograf)
Primary Outcome Measures
NameTimeMethod
Full tacrolimus AUC4 weeks

full tacrolimus AUC calculated from Tac measures before administration of drug and 1.5, 2, 4, 6, 8, 12, 13.5, 14, 16, 20, 24 hours after administration of drug at the time point of 2 weeks (14±7 days) after end of build-up period for each patient under both treatments within two time periods with each a length of 4 weeks

Secondary Outcome Measures
NameTimeMethod
Pharmacodynamic analysis4 weeks

Assessment of efficacy in terms of residual expression of NFAT regulated genes, expressed as % of expression at C0 (time point before drug administration set at 100%) at 1.5, 2, 4, 6, 8, 12, 13.5, 14, 16, 20, 24 hours after administration of drug at the time point of 2 weeks (14±7 days) after end of build-up period for each patient under both treatments within two time periods with each a length of 4 weeks

eGFR (CKiD formula)4 weeks

eGFR (CKiD formula) comparing the two study phases

Pharmacogenetic analysis4 weeks

Number of patients with SNPs in selected genes (CYP3A4, CYP3A5, ABCD1)

Taxonomy of the gut microbiome10 weeks

Taxonomy of the gut microbiome using metagenomic sequencing

Tacrolimus trough levels4 weeks

Tacrolimus trough levels in ng/mL, compared intra- and interindividually.

Number of adverse events or toxicity per patient10 weeks

Special attention is taken e.g. towards metabolic (elevated concentration of blood glucose, fat), hematopoetic (cell counts), neurological (tremor, headache), renal (change in glomerular filtration rate), gastrointestinal (diarrhea, nausea), hepatic (cholestasis, elevated transaminases, blood clotting disorder), elevated blood pressure.

Doses of prolonged-release tacrolimus4 weeks

Doses of prolonged-release tacrolimus (Envarsus®) in ng/mL.

Number of patients with adverse event or toxicity10 weeks

Cumulative dosage and signs of tacrolimus toxicity and adverse events. Potentially tacrolimus associated adverse events and toxicity are recorded individually and compared with individual tacrolimus AUCs. Special attention is taken e.g. towards metabolic (elevated concentration of blood glucose, fat), hematopoetic (cell counts), neurological (tremor, headache), renal (change in glomerular filtration rate), gastrointestinal (diarrhea, nausea), hepatic (cholestasis, elevated transaminases, blood clotting disorder), elevated blood pressure.

Treatment failure rate10 weeks

composite endpoint: any patient who experienced death, graft failure, BPAR or lost to follow-up

limited sampling strategy (LSS)4 weeks

LSS driven 24h-AUC estimation

Gut microbial metabolism10 weeks

Functional assessment of the gut microbiome using LC-MS based metabolomics

Trial Locations

Locations (4)

University Hospital of Essen, Pediatrics II

🇩🇪

Essen, Germany

University Hospital Cologne, Pediatrics

🇩🇪

Cologne, Germany

University Hospital of Hamburg-Eppendorf

🇩🇪

Hamburg, Germany

University Hospital of Heidelberg

🇩🇪

Heidelberg, Germany

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